Monday, May 21, 2018

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.


Signs of improvement with Duchenne gene therapy

A gene therapy from Solid Biosciences seems to be improving the symptoms of a teenager with Duchenne muscular dystrophy, the boy's father tells STAT's Adam Feuerstein

James Bradley, a police officer in Abbeville, Ala., says his son has seen marked improvement with the experimental therapy. “Braxton threw a softball overhand for the first time in his life,” he said. “In the past couple of weeks, he’s been able to move his outer extremities more efficiently and quickly.”

It’s just a single case report, and even Bradley concedes that it will take time and more patients to determine objectively if SGT-001 is the Duchenne cure he wants it to be. But his updates on the case — in a closed Facebook group  — are already playing a part in easing investor concerns about the safety of the Solid treatment.

Read more.

A financial roadblock for ALS patients

The FDA approved an ALS drug — Radicava —  just about two years ago, but some insurers are still not footing the $145,000 bill. So patients are seeing their disease worsen, without access to a drug that might improve their quality of life. 

The story of the drug, STAT's Ed Silverman writes, is a cautionary tale — one in which a costly medication can give rise to misplaced expectations and problems can be compounded by well-intentioned regulators trying to help patients whose medical needs are going unmet.

“I’m getting slower and weaker. It’s becoming harder to shampoo my hair or use a pencil. My disease is progressing slowly, but it is progressing,” one ALS patient told STAT. “I think the drug would make it possible to be with my family longer. So, it’s frustrating."

Read more. 

A bit of a misread

thanks for the deep insight, forbes. (twitter)

President Trump retweeted Damian's story about Amgen's lower-than-expected pricing of its new migraine drug, Aimovig. The only issue is, Steve Forbes — CEO of Forbes Media — misinterpreted things a bit. For one, it's unlikely that Trump's recent pricing speech inspired Amgen to price the drug 30 percent below projections. These decisions take time. 

But it's a sign that the White House has co-opted Amgen's long-considered decision to be a political victory, which has us wondering: What will they say about the next $300,000 rare disease drug?


There's a Black Mirror episode in here somewhere

More unsolved murders are getting another look, thanks to the kind of DNA detective work used in the case of the Golden State Killer. Police now have a suspect in the killing of a Canadian couple back in 1987: Investigators say they matched DNA taken from the crime site with genetic information available on the genealogy site GEDMatch, and zeroed in on truck driver William Earl Talbott II as the culprit. 

This new form of DNA sleuthing is playing a role in helping to crack more and more mysteries. But we do continue to wonder about the implications for genetic privacy: In this case, detectives tailed Talbott for days before collecting DNA from a discarded cup of his. 

More reads

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